Neuroprotection and neuroregeneration triggered through the FUS-induced opening of the blood-brain barrier in a Parkinson’s mouse model

Over the past decade, numerous small- and large-molecule products have been developed for treatment of neurodegenerative diseases with mixed success. When administered systemically in vivo, the blood-brain barrier (BBB) inhibits their delivery to the regions affected by those diseases. A successful drug delivery system requires transient, localized, and noninvasive targeting of a specific tissue region as provided by Focused Ultrasound (FUS). Neurturin (NTN) is a neurotrophic factor that has demonstrated to have neuroprotective and regenerative effects on dopaminergic neurons in vivo using invasive drug delivery methods. Utilizing recombinant adeno-associated virus (rAAV), therapeutic genes can be delivered to the brain for long-lasting treatments. In this study, we investigate the neuroprotective and neuroregenerative effects of non-invasively delivered rAAV-GDNF vectors and NTN in a PD mouse model, respectively.